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I3

ProtectomiR therapy

Prof. Peter Ferdinandy, Pharmahungary Group; Dr Zoltán Varga , Pharmahungary Group ; Dr Anikó Görbe, Pharmahungary Group

Pharmahungary Group


Challenge

Acute myocardial infarction (aMI) is an unmet medical need with currently no medication on the market. Only symptomatic drug treatment is available to patients with aMI, no curative treatment is on the market. Pharmahungary Group is developing a novel, IP protected in-house R&D project termed “ProtectomiR” therapy with the aim of curative treatment for ischemic heart diseases including e.g. aMI and heart failure.


Technology

ProtectomiR in-house R&D project is based on animal models of endogenous cardioprotection. Drug targets were selected by a currently available unbiased approach for target identification, i.e. transcriptomics. By transcriptomics approach, we have identified several cardioportective miRNA compounds that mimic endogenous cardioprotection. We termed these miRNAs ProtectomiRs. ProtectomiRs are miRNA compounds for therapeutic use for cytoprotection in ischemic diseases, including e.g. ischemic cardiac diseases such as aMI.


Commercial Opportunity

Ischemic heart disease including aMI and heart failure is still the leading causes of death worldwide with over 8.5 million deaths/year. The global myocardial infarction treatment market was estimated to be over 12 billion USD with expected to reach over 17 billion USD by 2022. This market estimation is for symptomatic drug treatment as no curative treatment is available on the market.


Development Status

Preclinical development plan prepared by worldwide opinion leader Pharmahungary Group scientific management team. Preclinical testing in small animals scheduled for Q1 2019. Planned timeline to reach final proof-of-concept in large animal models is up to 24 months and up to 36 months to start Phase I clinical trial.


Patent Situation

Patented technology: „Compounds for treatment of ischemic injury”, WO_2013/057527. Patents granted in USA and EU, currently in national phase in China. We are running large animal studies to identify further ProtectomiRs for patent family extension.


Further Reading

Varga VZ, et al.: MicroRNAs associated with ischemia-reperfusion injury and cardioprotection by ischemic pre- and postconditioning: protectomiRs. Am J Physiol Heart Circ Physiol. 2014 Jul 15;307(2):H216-27.

Perrino et al.: Epigenomic and transcriptomic approaches in the post-genomic era: path to novel targets for diagnosis and therapy of the ischaemic heart? Position Paper of the European Society of Cardiology Working Group on Cellular Biology of the Heart. Cardiovasc Res. 2017 Jun 1;113(7):725-736.


 

ProtectomiR therapy